Effects on Growth and Metabolism of Growth Hormone Treatment for 3 Years in 36 Children with Prader-Willi Syndrome
Effects on Growth and Metabolism of Growth Hormone Treatment for 3 Years in 36 Children with Prader-Willi Syndrome
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DOI：
10.1159/000319709
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发表时间：
2011-01-01
影响因子：
3.2
通讯作者：
Polak, M.
中科院分区：
文献类型：
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作者：
Colmenares, A.;Pinto, G.;Polak, M.
背景/目的：Prader-Willi综合征(PWS)是一种复杂的遗传性疾病，其多种表现包括肥胖和矮小。糖尿病、骨质疏松症和脊柱侧弯很常见。我们评估了人生长激素(HGH)的作用。方法：采用前瞻性队列研究方法，对36名1~15岁遗传确诊的PWS患儿进行为期36个月的hGH治疗(平均剂量0.033+/-0.006 mg/kg/d)。在基线和每年一次，我们评估生长、胰岛素样生长因子-1(IGF-1)、身体成分、骨密度(BMD)、糖耐量、血脂和脊柱X线片。结果：3年间身高增加1.2SD分。瘦体重在每个治疗年度都有显著增加。体脂总量在第1年和第2年分别下降了5.42%和1.17%。治疗过程中骨密度无明显变化。胰岛素样生长因子-1和胰岛素抵抗的稳态模型评估指数升高，基线时22.7%的患者存在糖耐量异常，3年时为0%。所有患者均未患糖尿病。他们的血脂状况有所改善。基线时27.8%的患者存在脊柱侧弯，3年时47.2%的患者存在脊柱侧弯。结论：生长激素治疗对儿童PWS的生长发育和体成分有多方面的有益影响。耐受性良好，葡萄糖代谢有所改善，尽管IGF-1水平和胰岛素抵抗参数应该密切监测。脊柱侧弯的高发生率需要儿科整形外科医生进行监测。版权所有(C)2010 S.Karger AG，巴塞尔
Background/Aims: Prader-Willi syndrome (PWS) is a complex genetic disorder whose many manifestations include obesity and short stature. Diabetes, osteoporosis, and scoliosis are common. We evaluated the effects of human growth hormone (hGH). Methods: A prospective cohort study of 36 children (1-15 years of age) with genetically confirmed PWS who were given hGH (mean dose 0.033 +/- 0.006 mg/kg/day) for 36 months. At baseline and once yearly, we evaluated growth, insulin-like growth factor-1 (IGF-1), body composition, bone mineral density (BMD), glucose tolerance, serum lipids, and spinal radiographs. Results: Height gain over the 3-year period was 1.2 SD score. Lean body mass increased significantly during each treatment year. Total body fat decreased by 5.42 and 1.17% in the 1st and 2nd years, respectively. BMD remained unchanged during therapy. IGF-1 and homeostasis model assessment index of insulin resistance increased, and glucose intolerance was found in 22.7% of patients at baseline and 0% at 3 years. None of the patients had diabetes.Their lipid profile improved. Scoliosis was present in 27.8% of the patients at baseline and 47.2% at 3 years. Conclusion: GH treatment in children with PWS has multiple beneficial effects on growth and body composition. Tolerance is good, with an improvement in glucose metabolism, although IGF-1 levels and insulin resistance parameters should be monitored closely. The high rate of scoliosis warrants monitoring by a pediatric orthopedic surgeon. Copyright (C) 2010 S. Karger AG, Basel